uniQure N.V. 10-Q

Item 1. Financial Statements uniQure N.V. ​ UNAUDITED CONSOLIDATED BALANCE SHEETS ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2020 2019 ​ ​ (in thousands, except share and per share amounts) Current assets ​ ​ ​ ​ ​ ​ Cash and cash equivalents ​ $ 314,265 ​ $ 377,793 Accounts receivable and accrued income from related party ​ ​ 222 ​ ​ 947 Prepaid expenses ​ ​ 4,082 ​ ​ 4,718 Other current assets ​ ​ 1,066 ​ ​ 748 Total current assets ​ ​ 319,635 ​ ​ 384,206 Non-current assets ​ ​ ​ ​ ​ ​ Property, plant and equipment, net of accumulated depreciation of $ 31.3 million as of June 30, 2020 and $ 28.6 million as of December 31, 2019, respectively ​ ​ 29,301 ​ ​ 28,771 Operating lease right-of-use assets ​ ​ 26,139 ​ ​ 26,797 Intangible assets, net ​ ​ 7,087 ​ ​ 5,427 Goodwill ​ ​ 496 ​ ​ 496 Restricted cash ​ ​ 2,683 ​ ​ 2,933 Total non-current assets ​ ​ 65,706 ​ ​ 64,424 Total assets ​ $ 385,341 ​ $ 448,630 Current liabilities ​ ​ ​ ​ ​ ​ Accounts payable ​ $ 4,942 ​ $ 5,681 Accrued expenses and other current liabilities ​ ​ 13,250 ​ ​ 12,457 Current portion of operating lease liabilities ​ ​ 5,495 ​ ​ 5,865 Current portion of deferred revenue ​ ​ 6,153 ​ ​ 7,627 Total current liabilities ​ ​ 29,840 ​ ​ 31,630 Non-current liabilities ​ ​ ​ ​ ​ ​ Long-term debt ​ ​ 35,373 ​ ​ 36,062 Operating lease liabilities, net of current portion ​ ​ 30,279 ​ ​ 31,133 Deferred revenue, net of current portion ​ ​ 23,048 ​ ​ 23,138 Derivative financial instruments related party ​ ​ 832 ​ ​ 3,075 Other non-current liabilities ​ ​ 464 ​ ​ 534 Total non-current liabilities ​ ​ 89,996 ​ ​ 93,942 Total liabilities ​ ​ 119,836 ​ ​ 125,572 Commitments and contingencies ​ ​ ​ ​ ​ ​ Shareholders' equity ​ ​ ​ ​ ​ ​ Ordinary shares, € 0.05 par value: 60,000,000 shares authorized at June 30, 2020 and December 31, 2019 and 44,444,405 and 43,711,954 ordinary shares issued and outstanding at June 30, 2020 and December 31, 2019, respectively ​ ​ 2,692 ​ ​ 2,651 Additional paid-in-capital ​ ​ 1,000,389 ​ ​ 986,803 Accumulated other comprehensive loss ​ ​ ( 7,319 ) ​ ​ ( 6,689 ) Accumulated deficit ​ ​ ( 730,257 ) ​ ​ ( 659,707 ) Total shareholders' equity ​ ​ 265,505 ​ ​ 323,058 Total liabilities and shareholders' equity ​ $ 385,341 ​ $ 448,630 ​ The accompanying notes are an integral part of these unaudited consolidated financial statements. ​ ​ ​ ​ ​ 2 Table of Contents uniQure N.V. ​ UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended June 30, ​ Six months ended June 30, ​ 2020 2019 2020 2019 ​ ​ (in thousands, except share and per share amounts) ​ (in thousands, except share and per share amounts) License revenues from related party ​ ​ 1,530 ​ ​ 2,108 ​ ​ 1,577 ​ ​ 2,665 Collaboration revenues from related party ​ ​ 5 ​ ​ 366 ​ ​ 62 ​ ​ 945 Total revenues ​ ​ 1,535 ​ ​ 2,474 ​ ​ 1,639 ​ ​ 3,610 Operating expenses: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Research and development expenses ​ ​ ( 28,401 ) ​ ​ ( 24,154 ) ​ ​ ( 54,414 ) ​ ​ ( 44,691 ) Selling, general and administrative expenses ​ ​ ( 11,511 ) ​ ​ ( 7,870 ) ​ ​ ( 20,583 ) ​ ​ ( 15,937 ) Total operating expenses ​ ​ ( 39,912 ) ​ ​ ( 32,024 ) ​ ​ ( 74,997 ) ​ ​ ( 60,628 ) Other income ​ ​ 669 ​ ​ 566 ​ ​ 1,526 ​ ​ 879 Other expense ​ ​ ( 500 ) ​ ​ ( 347 ) ​ ​ ( 839 ) ​ ​ ( 696 ) Loss from operations ​ ​ ( 38,208 ) ​ ​ ( 29,331 ) ​ ​ ( 72,671 ) ​ ​ ( 56,835 ) Interest income ​ ​ 81 ​ ​ 728 ​ ​ 903 ​ ​ 1,170 Interest expense ​ ​ ( 970 ) ​ ​ ( 937 ) ​ ​ ( 1,945 ) ​ ​ ( 1,894 ) Foreign currency (losses) / gains, net ​ ​ ( 3,645 ) ​ ​ ( 1,252 ) ​ ​ 957 ​ ​ 1,022 Other non-operating gains / (losses), net ​ ​ 191 ​ ​ ( 607 ) ​ ​ 2,206 ​ ​ ( 2,634 ) Net loss ​ $ ( 42,551 ) ​ $ ( 31,399 ) ​ $ ( 70,550 ) ​ $ ( 59,171 ) Other comprehensive income / (loss): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Foreign currency translation adjustments ​ ​ 4,647 ​ ​ 1,209 ​ ​ ( 630 ) ​ ​ ( 1,259 ) Total comprehensive loss ​ $ ( 37,904 ) ​ $ ( 30,190 ) ​ $ ( 71,180 ) ​ $ ( 60,430 ) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Basic and diluted net loss per ordinary share ​ $ ( 0.96 ) ​ $ ( 0.83 ) ​ $ ( 1.59 ) ​ $ ( 1.57 ) Weighted average shares used in computing basic and diluted net loss per ordinary share ​ ​ 44,387,463 ​ ​ 37,824,928 ​ ​ 44,333,460 ​ ​ 37,750,961 ​ ​ The accompanying notes are an integral part of these unaudited consolidated financial statements. ​ 3 Table of Contents uniQure N.V. ​ UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITY FOR THE THREE-MONTH PERIOD ENDED JUNE 30 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Accumulated ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Additional ​ other ​ ​ ​ ​ Total ​ Ordinary shares ​ paid-in ​ comprehensive ​ Accumulated ​ shareholders’ ​ No. of shares Amount capital (loss)/income deficit equity ​ (in thousands, except share and per share amounts) Balance at March 31, 2019 37,763,842 ​ $ 2,322 ​ $ 727,795 ​ $ ( 9,727 ) ​ $ ( 563,278 ) ​ $ 157,112 Loss for the period — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 31,399 ) ​ ​ ( 31,399 ) Other comprehensive income — ​ ​ — ​ ​ — ​ ​ 1,209 ​ ​ — ​ ​ 1,209 Hercules warrants exercise — ​ ​ — ​ ​ — ​ ​ — ​ ​ — ​ ​ - Exercise of share options 52,847 ​ ​ 4 ​ ​ 454 ​ ​ — ​ ​ — ​ ​ 458 Restricted and performance share units distributed during the period 21,400 ​ ​ 1 ​ ​ ( 1 ) ​ ​ — ​ ​ — ​ ​ — Share-based compensation expense — ​ ​ — ​ ​ 4,592 ​ ​ — ​ ​ — ​ ​ 4,592 Issuance of ordinary shares relating to employee stock purchase plan 1,744 ​ ​ — ​ ​ 84 ​ ​ — ​ ​ — ​ ​ 84 Balance at June 30, 2019 37,839,833 ​ $ 2,327 ​ $ 732,924 ​ $ ( 8,518 ) ​ $ ( 594,677 ) ​ $ 132,056 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Balance at March 31, 2020 44,299,596 ​ $ 2,683 ​ $ 992,136 ​ $ ( 11,966 ) ​ $ ( 687,706 ) ​ $ 295,147 Loss for the period — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 42,551 ) ​ ​ ( 42,551 ) Other comprehensive income — ​ ​ — ​ ​ — ​ ​ 4,647 ​ ​ — ​ ​ 4,647 Exercise of share options 139,178 ​ ​ 9 ​ ​ 2,461 ​ ​ — ​ ​ — ​ ​ 2,470 Restricted and performance share units distributed during the period 4,427 ​ ​ — ​ ​ — ​ ​ — ​ ​ — ​ ​ — Share-based compensation expense — ​ ​ — ​ ​ 5,723 ​ ​ — ​ ​ — ​ ​ 5,723 Issuance of ordinary shares relating to employee stock purchase plan 1,204 ​ ​ — ​ ​ 69 ​ ​ — ​ ​ — ​ ​ 69 Balance at June 30, 2020 44,444,405 ​ $ 2,692 ​ $ 1,000,389 ​ $ ( 7,319 ) ​ $ ( 730,257 ) ​ $ 265,505 ​ The accompanying notes are an integral part of these unaudited consolidated financial statements. ​ ​ ​ ​ ​ ​ ​ ​ ​ 4 Table of Contents uniQure N.V. ​ UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITY FOR THE SIX-MONTH PERIOD ENDED JUNE 30 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Accumulated ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Additional ​ other ​ ​ ​ ​ Total ​ Ordinary shares ​ paid-in ​ comprehensive ​ Accumulated ​ shareholders’ ​ No. of shares Amount capital (loss)/income deficit equity ​ (in thousands, except share and per share amounts) Balance at December 31, 2018 37,351,653 ​ $ 2,299 ​ $ 720,072 ​ $ ( 7,259 ) ​ $ ( 535,506 ) ​ $ 179,606 Loss for the period — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 59,171 ) ​ ​ ( 59,171 ) Other comprehensive loss — ​ ​ — ​ ​ — ​ ​ ( 1,259 ) ​ ​ — ​ ​ ( 1,259 ) Hercules warrants exercise 37,175 ​ ​ 2 ​ ​ 1,271 ​ ​ — ​ ​ — ​ ​ 1,273 Exercise of share options 236,654 ​ ​ 14 ​ ​ 2,542 ​ ​ — ​ ​ — ​ ​ 2,556 Restricted and performance share units distributed during the period 209,481 ​ ​ 12 ​ ​ ( 12 ) ​ ​ — ​ ​ — ​ ​ — Share-based compensation expense — ​ ​ — ​ ​ 8,886 ​ ​ — ​ ​ — ​ ​ 8,886 Issuance of ordinary shares relating to employee stock purchase plan 4,870 ​ ​ — ​ ​ 165 ​ ​ — ​ ​ — ​ ​ 165 Balance at June 30, 2019 37,839,833 ​ $ 2,327 ​ $ 732,924 ​ $ ( 8,518 ) ​ $ ( 594,677 ) ​ $ 132,056 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Balance at December 31, 2019 43,711,954 ​ $ 2,651 ​ $ 986,803 ​ $ ( 6,689 ) ​ $ ( 659,707 ) ​ $ 323,058 Loss for the period — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 70,550 ) ​ ​ ( 70,550 ) Other comprehensive loss — ​ ​ — ​ ​ — ​ ​ ( 630 ) ​ ​ — ​ ​ ( 630 ) Exercise of share options 203,940 ​ ​ 12 ​ ​ 3,390 ​ ​ — ​ ​ — ​ ​ 3,402 Restricted and performance share units distributed during the period 525,506 ​ ​ 29 ​ ​ ( 29 ) ​ ​ — ​ ​ — ​ ​ — Share-based compensation expense — ​ ​ — ​ ​ 10,078 ​ ​ — ​ ​ — ​ ​ 10,078 Issuance of ordinary shares relating to employee stock purchase plan 3,005 ​ ​ — ​ ​ 147 ​ ​ — ​ ​ — ​ ​ 147 Balance at June 30, 2020 44,444,405 ​ $ 2,692 ​ $ 1,000,389 ​ $ ( 7,319 ) ​ $ ( 730,257 ) ​ $ 265,505 ​ ​ The accompanying notes are an integral part of these unaudited consolidated financial statements. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 5 Table of Contents uniQure N.V. ​ UNAUDITED CONSOLIDATED STATEMENTS OF CASH FLOWS ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Six months ended June 30, ​ 2020 2019 ​ ​ ​ ​ (in thousands) Cash flows from operating activities ​ ​ ​ ​ ​ ​ ​ Net loss ​ $ ( 70,550 ) ​ $ ( 59,171 ) ​ Adjustments to reconcile net loss to net cash used in operating activities: ​ ​ ​ ​ ​ ​ ​ Depreciation and amortization ​ ​ 3,505 ​ ​ 3,217 ​ Share-based compensation expense ​ ​ 10,078 ​ ​ 8,886 ​ Change in fair value of derivative financial instruments ​ ​ ( 2,206 ) ​ ​ 2,634 ​ Unrealized foreign exchange gains ​ ​ ( 908 ) ​ ​ ( 386 ) ​ Changes in operating assets and liabilities: ​ ​ ​ ​ ​ ​ ​ Accounts receivable and accrued income, prepaid expenses and other current assets ​ ​ 1,050 ​ ​ ( 2,779 ) ​ Accounts payable ​ ​ ( 981 ) ​ ​ 283 ​ Accrued expenses, other liabilities and operating leases ​ ​ ( 1,332 ) ​ ​ ( 658 ) ​ Deferred revenue ​ ​ ( 1,600 ) ​ ​ ( 2,683 ) ​ Net cash used in operating activities ​ ​ ( 62,944 ) ​ ​ ( 50,657 ) ​ Cash flows from investing activities ​ ​ ​ ​ ​ ​ ​ Purchases of intangible assets ​ ​ ( 2,214 ) ​ ​ ( 996 ) ​ Purchases of property, plant and equipment ​ ​ ( 2,392 ) ​ ​ ( 1,432 ) ​ Net cash used in investing activities ​ ​ ( 4,606 ) ​ ​ ( 2,428 ) ​ Cash flows from financing activities ​ ​ ​ ​ ​ ​ ​ Proceeds from issuance of shares related to employee stock option and purchase plans ​ ​ 3,549 ​ ​ 2,721 ​ Proceeds from exercise of warrants ​ ​ - ​ ​ 500 ​ Net cash generated from financing activities ​ ​ 3,549 ​ ​ 3,221 ​ Currency effect on cash, cash equivalents and restricted cash ​ ​ 223 ​ ​ ( 443 ) ​ Net decrease in cash, cash equivalents and restricted cash ​ ​ ( 63,778 ) ​ ​ ( 50,307 ) ​ Cash, cash equivalents and restricted cash at beginning of period ​ ​ 380,726 ​ ​ 237,342 ​ Cash, cash equivalents and restricted cash at the end of period ​ $ 316,948 ​ $ 187,035 ​ Cash and cash equivalents ​ $ 314,265 ​ $ 184,095 ​ Restricted cash related to leasehold and other deposits ​ ​ 2,683 ​ ​ 2,940 ​ Total cash, cash equivalents and restricted cash ​ $ 316,948 ​ $ 187,035 ​ Supplemental cash flow disclosures: ​ ​ ​ ​ ​ ​ ​ Cash paid for interest ​ $ ( 2,545 ) ​ $ ( 1,544 ) ​ Non-cash increases in accounts payables and accrued expenses and other current liabilities related to purchases of intangible assets and property, plant and equipment ​ $ 1,021 ​ $ 473 ​ ​ The accompanying notes are an integral part of these unaudited consolidated financial statements. ​ ​ 6 Table of Contents 1 General business information ​ uniQure (the “Company”) was incorporated on January 9, 2012 as a private company with limited liability ( besloten vennootschap met beperkte aansprakelijkheid ) under the laws of the Netherlands. The Company is a leader in the field of gene therapy and seeks to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. The Company’s business was founded in 1998 and was initially operated through its predecessor company, Amsterdam Molecular Therapeutics (AMT) Holding N.V (“AMT”). In 2012, AMT undertook a corporate reorganization, pursuant to which uniQure B.V. acquired the entire business and assets of AMT and completed a share-for-share exchange with the shareholders of AMT. Effective February 10, 2014, in connection with its initial public offering, the Company converted into a public company with limited liability ( naamloze vennootschap ) and changed its legal name from uniQure B.V. to uniQure N.V. ​ The Company is registered in the trade register of the Chamber of Commerce ( Kamer van Koophandel ) in Amsterdam, the Netherlands under number 54385229. The Company’s headquarters are in Amsterdam, the Netherlands, and its registered office is located at Paasheuvelweg 25, Amsterdam 1105 BP, the Netherlands and its telephone number is +31 20 240 6000. ​ The Company’s ordinary shares are listed on the Nasdaq Global Select Market and trade under the symbol “QURE”. ​ 2 Summary of significant accounting policies ​ 2.1 Basis of preparation ​ The Company prepared these unaudited consolidated financial statements in compliance with generally accepted accounting principles in the United States of America (“U.S. GAAP”) and applicable rules and regulations of the SEC regarding interim financial reporting. Any reference in these notes to applicable guidance is meant to refer to authoritative U.S. GAAP as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Update (“ASU”) of the Financial Accounting Standards Board (“FASB”). ​ The unaudited consolidated financial statements are presented in U.S. dollars, except where otherwise indicated. Transactions denominated in currencies other than U.S. dollars are presented in the transaction currency with the U.S. dollar amount included in parenthesis, converted at the foreign exchange rate as of the transaction date. ​ 2.2 Unaudited interim financial information ​ The interim financial statements and related disclosures are unaudited, have been prepared on the same basis as the annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for a fair statement of the financial position, results of operations and changes in financial position for the period presented. ​ Certain information and footnote disclosures normally included in annual financial statements prepared in accordance with U.S. GAAP have been omitted. The results of operations for the six months ended June 30, 2020, are not necessarily indicative of the results to be expected for the full year ending December 31, 2020 or for any other future year or interim period. The accompanying financial statements should be read in conjunction with the audited financial statements and the related notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2019, filed with the SEC on March 2, 2020 . ​ 2.3 Use of estimates ​ The preparation of the financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities at the date of the financial statements and reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates. ​ 7 Table of Contents 2.4 Accounting policies ​ The principal accounting policies applied in the preparation of these unaudited consolidated financial statements are described in the Company’s audited financial statements as of and for the year ended December 31, 2019, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2019, filed with the SEC on March 2, 2020 . There have been no material changes in the Company’s significant accounting policies during the six months ended June 30, 2020. ​ 2.5 Recent accounting pronouncements ​ There have been no new accounting pronouncements or changes to accounting pronouncements during the six months ended June 30, 2020, as compared to the recent accounting pronouncements described in Note 2.3.23 of the Company’s Annual Report on Form 10-K for the year ended December 31, 2019 , which could be expected to materially impact the Company’s unaudited consolidated financial statements. The Company has adopted ASU 2018-13, Fair Value Measurement (Topic 820) and additional disclosures related to significant unobservable inputs have been included within footnote 4 Fair value measurement. ​ 3 Collaboration arrangements and concentration of credit risk ​ CSL Behring collaboration ​ On June 24, 2020, uniQure biopharma B.V., a wholly-owned subsidiary of uniQure N.V., entered into a commercialization and license agreement (the “CSL Behring Agreement”) with CSL Behring LLC (“CSL Behring”) providing CSL Behring exclusive global rights to etranacogene dezaparvovec, the Company’s investigational gene therapy for patients with hemophilia B (the “Product”). ​ Under the terms of the CSL Behring Agreement, the Company will receive a $ 450 million upfront cash payment upon the closing of the CSL Behring Agreement and be eligible to receive up to $ 1.6 billion in payments based on regulatory and commercial milestones. The Company will also be eligible to receive tiered double-digit royalties in a range of up to a low-twenties percent of net sales of the Product based on sales thresholds. Pursuant to the CSL Behring Agreement, the Company will be responsible for the completion of the HOPE-B clinical trial, manufacturing process validation, and the manufacturing supply of Product until such time that these capabilities may be transferred to CSL Behring or its designated contract manufacturing organization. Pursuant to a development and commercial supply agreement, the Company will supply Product to CSL Behring at an agreed-upon price per the contract. The Company and CSL Behring executed the development and commercial supply agreement simultaneously with the CSL Behring Agreement. Certain provisions will not become effective until after the Company receives regulatory approval to close the transaction. Clinical development and regulatory activities performed by the Company pursuant to the CSL Behring Agreement will be reimbursed by CSL Behring. CSL Behring will be responsible for global regulatory submissions and commercialization requirements for Product. Closing of the CSL Behring Agreement is contingent on the successful completion of reviews under antitrust laws in the United States, Australia, and the United Kingdom, which has not occurred to date. Closing of the transaction is dependent on the timing, extent, and result of the regulatory review process. The Company does not believe that the consummation of the transaction will result in a violation of any applicable antitrust laws. However, there can be no assurance that a challenge on antitrust grounds will not be made, or if such a challenge is made, what the result would be. In accordance with its existing license and other agreements the Company is contractually required to pay in total a low to high single digit percentage of any upfront payment to its licensors and financial advisor (“License Fees”). As of June 30, 2020, the Company concluded it has no enforceable right to receive any of the upfront payment, the regulatory and sales milestone payments or the royalties (together “CSL License Revenue”) that it may receive in accordance with the CSL Behring Agreement, as all payments are contingent upon the successful completion of reviews under antitrust laws in the United States, Australia and the United Kingdom. Therefore, the Company determined it will not recognize any revenue in relation to the CSL License Revenue, in accordance with ASC 606. 8 Table of Contents The Company determined that in accordance with Dutch taw law it will recognize the CSL Behring License Revenue as well as the License Fees as taxable results as of the date on which the Company is contractually entitled to receive (or obligated to make) a payment under the CSL Behring Agreement. The Company expects to continue to incur taxable losses in the Netherlands except for the period in which it receives the $ 450 million upfront payment. In the event that the Company recognizes the $ 450 million upfront payment in 2020, such payment will be subject to Dutch corporate income tax at a rate of 25.0 %. Any CSL License Revenue including the upfront payment that the Company recognizes thereafter will be subject to Dutch corporate income tax at a rate of 21.7 %. However, the Company does not expect it will be required to pay any income taxes in the period in which it recognizes the $ 450 million upfront payment as taxable revenue as such payment is not expected to exceed the net operating losses the Company has carried forward in the Netherlands. The Company has historically recorded a full valuation allowance against its net deferred tax assets. Future taxable income or losses of the Company and a potential reversal of the valuation allowance will be impacted by a variety of factors, of which some are outside of the Company’s control. Such factors include the outcome and timing of the reviews under antitrust laws in the United States, Australia, and the United Kingdom, the change in the Dutch corporate income tax rate in 2021, additional net operating losses the Company may generate in relation to the CSL Behring Agreement, and the amount of License Fees the Company is required to pay. ​ The Company recognizes deferred tax assets to the extent that it determines that these assets are more likely than not to be realized. In making such a determination, the Company weighed all available positive and negative evidence, including future income projections from the CSL Behring Agreement, and concluded that it is more likely than not that the deferred tax assets will not be realized. Accordingly, the Company continued to record a full valuation allowance as of June 30, 2020. ​ Bristol-Myers Squibb collaboration ​ In May 2015, the Company entered into a collaboration and license agreement (the “BMS CLA”) and various related agreements with Bristol-Myers Squibb Company (“BMS”) that provide BMS with exclusive access to the Company’s gene therapy technology platform for the research, development and commercialization of therapeutics aimed at multiple targets in cardiovascular and other diseases (“Collaboration Targets”). The initial four-year research term under the collaboration terminated on May 21, 2019. During the initial research term of the BMS CLA, the Company supported BMS in discovery, non-clinical, analytical and process development efforts in respect of the Collaboration Targets. For any Collaboration Targets that may be advanced, the Company will be responsible for manufacturing of clinical and commercial supplies using the Company’s vector technologies and industrial, proprietary insect-cell based manufacturing platform. BMS reimbursed the Company for all its research and development costs in support of the collaboration during the initial research term, and will lead development, regulatory and commercial activities for any Collaboration Targets that may be advanced. The BMS CLA provides that the Company and BMS may collaborate on up to ten Collaboration Targets in total. As of June 30, 2020, BMS has designated a total of four Collaboration Targets. In February 2019, BMS requested a one-year extension of the research term. In April 2019, following an assessment of the progress of this collaboration and the Company’s expanding proprietary programs, the Company notified BMS that the Company did not intend to agree to an extension of the research term but rather preferred to restructure or amend the collaboration to reduce or eliminate certain of the Company’s obligations under it. ​ The Company has agreed to certain restrictions on its ability to work independently of the collaboration, either directly or indirectly through any affiliate or third party, on certain programs that would be competitive with the collaboration programs. The Company is currently in discussions with BMS potentially to amend the BMS CLA and other related agreements following the expiration of the research term. It is currently uncertain whether a change to the BMS CLA will be agreed and, if agreed, what the specific terms of any such change may be. As a consequence, the Company has not taken into account the impact of such change, if any, on the timing of recognition of the prepaid License Revenue if and when the BMS CLA and other related agreements have been restructured or amended. The final resolution of these discussions may or may not result in material changes to the Company’s collaboration with BMS. The Company agreed, subject to certain conditions, to continue providing limited support of the pre-clinical Collaboration Targets, and any related costs will be reimbursed by BMS during these discussions. 9 Table of Contents The Company evaluated the BMS CLA and determined that its performance obligations are as follows: (i) Providing access to its technology and know-how in the field of gene therapy as well as actively contributing to the target selection, the collaboration as a whole, the development during the target selection, the pre-clinical and the clinical phase through participating in joint steering committee and other governing bodies (“License Revenue”); (ii) Providing pre-clinical Collaboration Target specific, non-clinical, analytical and process development services during the initial research term, which ended on May 21, 2019 (“Collaboration Revenue”); and (iii) Providing clinical and commercial manufacturing services for Collaboration Targets (“Manufacturing Revenue”). To date the Company has not generated any Manufacturing Revenue. ​ Amounts owed by BMS in relation the collaboration services are as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2020 2019 ​ ​ (in thousands) Bristol Myers Squibb ​ $ 222 ​ $ 947 Total ​ $ 222 ​ $ 947 ​ License Revenue The Company recognized $ 1.5 million and $ 1.6 million of License Revenue for the three and six months ended June 30, 2020, respectively, compared to $ 2.1 million and $ 2.7 million during the same periods in 2019 in relation to a $ 60.1 million upfront payment recorded on May 21, 2015, as well as $ 15.0 million received in relation to the designation of the second, third and fourth Collaboration Targets in August 2015 (together “Consideration”). The Company would be entitled to an aggregate $ 16.5 million in target designation payments upon the selection of the fifth through tenth Collaboration Targets. The Company would also be eligible to receive research, development and regulatory milestone payments of up to $ 254.0 million for a lead Collaboration Target and up to $ 217.0 million for each of the other selected Collaboration Targets, if defined milestones are achieved. The Company would include the variable consideration related to the selection of the fifth to tenth Collaboration Target, or any of the milestones, in the transaction price once it is considered probable that including these payments in the transaction price would not result in the reversal of cumulative revenue recognized. The Company would recognize significant amounts of License Revenue for services performed in prior periods if and when the Company considers this probable. Due to the significant uncertainty surrounding the development of gene-therapy product candidates and the dependence on BMS’s performance and decisions, the Company does not currently consider this probable. Additionally, the Company is eligible to receive net sales-based milestone payments and tiered mid-single to low double-digit royalties on product sales. The royalty term is determined on a licensed-product-by-licensed-product and country-by-country basis and begins on the first commercial sale of a licensed product in a country and ends on the expiration of the last to expire of specified patents or regulatory exclusivity covering such licensed product in such country or, with a customary royalty reduction, ten years after the first commercial sale if there is no such exclusivity. These revenues will be recognized when performance obligations are satisfied. The Company recognizes License Revenue over the expected performance period based on its measure of progress towards the completion of certain activities related to its services. The Company determines such progress by comparing activities performed at the end of each reporting period with total activities expected to be performed. The Company estimates total expected activities using a number of unobservable inputs, such as the probability of BMS designating additional targets, the probability of successfully completing each phase and estimated time required to provide services during the various development stages. If available, the Company uses product candidate-specific research and development plans. Alternatively, the Company assumes that completion of the pre-clinical phase requires an average of four years and that clinical development and commercial launch on average require 8.5 years. ​ 10 Table of Contents 4 Fair value measurement ​ The Company measures certain assets and liabilities at fair value, either upon initial recognition or for subsequent accounting or reporting. U.S. GAAP requires disclosure of methodologies used in determining the reported fair values, and establishes a hierarchy of inputs used when available. The three levels of the fair value hierarchy are described below: ● Level 1 - Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company can access at the measurement date. ● Level 2 - Valuations based on quoted prices for similar assets or liabilities in markets that are not active or models for which the inputs are observable, either directly or indirectly. ● Level 3 - Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and are unobservable. To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized as Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. ​ The carrying amount of cash and cash equivalents, accrued income from the related party, prepaid expenses, other assets, accounts payable, accrued expenses and other current liabilities reflected in the Consolidated balance sheets approximate their fair values due to their short-term maturities. The following table sets forth the Company’s assets and liabilities that are required to be measured at fair value on a recurring basis as of June 30, 2020, and December 31, 2019: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Quoted prices in active markets (Level 1) Significant other observable inputs (Level 2) Significant unobservable inputs (Level 3) Total Classification in Consolidated balance sheets ​ ​ ​ (in thousands) ​ ​ At December 31, 2019 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Assets: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash, cash equivalents and restricted cash ​ $ 380,726 ​ $ — ​ $ — ​ $ 380,726 ​ Cash and cash equivalents; restricted cash Total assets ​ $ 380,726 ​ $ — ​ $ — ​ $ 380,726 ​ ​ Liabilities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Derivative financial instruments - related party ​ ​ — ​ ​ — ​ ​ 3,075 ​ ​ 3,075 ​ ​ Total liabilities ​ $ — ​ $ — ​ $ 3,075 ​ $ 3,075 ​ ​ At June 30, 2020 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Assets: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash, cash equivalents and restricted cash ​ $ 316,948 ​ $ — ​ $ — ​ $ 316,948 ​ Cash and cash equivalents; restricted cash Total assets ​ $ 316,948 ​ $ — ​ $ — ​ $ 316,948 ​ ​ Liabilities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Derivative financial instruments - related party ​ $ — ​ $ — ​ $ 832 ​ $ 832 ​ ​ Total liabilities ​ $ — ​ $ — ​ $ 832 ​ $ 832 ​ ​ ​ Changes in Level 3 items during the six months ended June 30, 2020, are as follows: ​ ​ ​ ​ ​ ​ ​ Derivative ​ ​ financial ​ instruments ​ ​ (in thousands) Balance at December 31, 2019 ​ $ 3,075 Net gains recognized in profit or loss ​ ​ ( 2,206 ) Currency translation effects ​ ​ ( 37 ) Balance at June 30, 2020 ​ $ 832 ​ 11 Table of Contents ​ ​ ​ ​ BMS warrants ​ The Company issued derivative financial instruments related to its collaboration with BMS. The fair value of the BMS derivative financial instruments (“BMS warrants”) as of June 30, 2020, was $ 0.8 million compared to a fair value of $ 3.1 million as of December 31, 2019. Changes in the fair value of the BMS warrants are primarily impacted by changes in the Company’s share price, whereby a decrease in share price generally results in a decrease of the fair value. In addition, the Company revised certain unobservable inputs as well as valuation techniques in March 2020 which resulted in a further decrease in fair value of $ 0.7 million when compared to December 31, 2019. These BMS warrants are described in more detail below. ​ The Company granted BMS two warrants: ​ ● A warrant allowing BMS to purchase a specific number of the Company’s unregistered ordinary shares such that its ownership will equal 14.9 % immediately after such purchase (“1 st warrant”). The 1 st warrant can be exercised on the later of (i) the date on which the Company receives from BMS the Target Designation Fees (as defined in the BMS CLA) associated with the first six new targets (a total of seven Collaboration Targets); and (ii) the date on which BMS designates the sixth new target (the seventh Collaboration Target). ● A warrant allowing BMS to purchase a specific number of the Company’s unregistered ordinary shares such that its ownership will equal 19.9 % immediately after such purchase (“2 nd warrant” and together with the 1 st warrant, the “warrants”). The 2 nd warrant can be exercised on the later of (i) the date on which the Company receives from BMS the Target Designation Fees associated with the first nine new targets (a total of ten Collaboration Targets); and (ii) the date on which BMS designates the ninth new target (the tenth Collaboration Target). Pursuant to the terms of the BMS CLA, the exercise price in respect of each warrant is equal to the greater of (i) the product of (A) $ 33.84 and (B) a compounded annual growth rate of 10 % (or approximately $ 55.07 as of June 30, 2020) and (ii) the product of (A) 1.10 and (B) the VWAP for the 20 trading days ending on the date that is five trading days prior to the date of a notice of exercise delivered by BMS. ​ As of June 30, 2020, BMS had designated a total of four Collaboration Targets, and as such, the warrants were not exercisable. The Company estimated the exercise of warrants to occur within four and five years after the balance sheet date. ​ The significant unobservable inputs that the Company uses to develop Level 3 fair value measurements include the number of ordinary shares outstanding at the time of BMS’s warrant exercises. The number of such ordinary shares outstanding at the time of exercise determines the number of unregistered ordinary shares to be issued in connection with the BMS warrants. ​ The warrants can only be exercised following the occurrence of events contractually defined in the warrant agreements. The probability of the occurrence of these events represent another significant unobservable input used in the calculation of the fair value of the warrants. The Company estimates that the probability of the occurrence of events allowing BMS to exercise the 1 st warrant is within a range of 0 % to 44 % and between 0 % to 11 % with respect to the 2 nd warrant. The arithmetic averages related to these ranges are 24 % and 5 % for the 1 st and 2 nd warrant, respectively. ​ ​ ​ ​ ​ 12 Table of Contents 5 Right-of-use assets and lease liabilities ​ The Company’s most significant leases relate to office and laboratory space under the following operating lease agreements: ​ Lexington, Massachusetts / United States ​ In July 2013, the Company entered into a lease for a facility in Lexington, Massachusetts, United States. In November 2018, the term was expanded by five years from 2024 to June 2029. The lease is renewable for two subsequent five-year terms. Additionally, the lease was expanded to include an additional 30,655 square feet within the same facility and for the same term. The lease of the expansion space commenced on June 1, 2019. ​ Amsterdam / The Netherlands ​ In March 2016, the Company entered into a 16-year lease for a facility in Amsterdam, the Netherlands, and amended this agreement in June 2016. The lease for this facility terminates in February 2032, with an option to extend in increments of five-year periods. The lease contract includes variable lease payments related to annual increases in payments based on a consumer price index. ​ On December 1, 2017, the Company entered into an agreement to sub-lease three of the seven floors of its Amsterdam facility for a ten-year term ending on December 31, 2027, with an option for the sub-lessee to extend until December 31, 2031. In February 2020, the Company amended the agreement to sub-lease to take back one of the three floors effective March 1, 2020. The fixed lease payments to be received during the remaining term under the agreement to sub-lease amount to $ 6.4 million (EUR 5.7 million) as of June 30, 2020. ​ The table below presents the components of the Company’s lease costs for the periods indicated: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended June 30, Six months ended June 30, ​ 2020 2019 2020 2019 ​ ​ (in thousands) ​ (in thousands) Operating lease cost ​ $ 1,289 ​ $ 1,022 ​ $ 2,579 ​ $ 1,919 Variable lease cost ​ ​ 153 ​ ​ 101 ​ ​ 298 ​ ​ 182 Sublease income ​ ​ ( 209 ) ​ ​ ( 264 ) ​ ​ ( 457 ) ​ ​ ( 532 ) Total lease cost ​ $ 1,233 ​ $ 859 ​ $ 2,420 ​ $ 1,569 ​ ​ The table below presents the lease-related assets and liabilities recorded on the Consolidated balance sheets for the periods indicated. ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2020 ​ 2019 ​ ​ (in thousands) Assets ​ ​ ​ ​ ​ ​ Operating lease right-of-use assets ​ $ 26,139 ​ ​ 26,797 Liabilities ​ ​ ​ ​ ​ ​ Current ​ ​ ​ ​ ​ ​ Current operating lease liabilities ​ ​ 5,495 ​ ​ 5,865 Non-current ​ ​ ​ ​ ​ ​ Non-current operating lease liabilities ​ ​ 30,279 ​ ​ 31,133 Total lease liabilities ​ $ 35,774 ​ ​ 36,998 ​ 13 Table of Contents Other information ​ The weighted-average remaining lease term as of June 30, 2020 is 9.8 years, compared to 10.3 years as of December 31, 2019 and the weighted-average discount rate as of June 30, 2020 is 11.33 %, compared to 11.33 % as of December 31, 2019. The Company derived the weighted-average discount rate, adjusted for differences such as in the term and payment patterns, from the Company’s loan from Hercules Capital which was refinanced immediately prior to the January 1, 2019 adoption date in December 2018. ​ Cash paid for amounts included in the measurement of lease liabilities for the periods indicated below was as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended June 30, Six months ended June 30, ​ 2020 2019 2020 2019 ​ (in thousands) ​ (in thousands) Operating cash flows for operating leases $ 1,731 ​ $ 603 ​ $ 3,155 ​ $ 2,016 ​ The Company did not obtain any right-of-use assets in exchange for lease obligations in the three and six months ended June 30, 2020. Besides the initial recognition of operating right-of-use assets of $ 19.0 million upon adoption of the new lease standards on January 1, 2019, the Company obtained $ 8.6 million of additional right-of-use assets in exchange for lease obligations in the three and six months ended June 30, 2019. ​ Undiscounted cash flows ​ The table below reconciles the undiscounted cash flows as of June 30, 2020, for each of the first five years and the total of the remaining years to the operating lease liabilities recorded on the Consolidated balance sheet as of June 30, 2020. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Lexington ​ Amsterdam 1) ​ Other 1) ​ Total ​ (in thousands) 2020 (six months remaining) $ 1,697 ​ $ 947 ​ $ 61 ​ $ 2,705 2021 ​ 3,455 ​ ​ 1,894 ​ ​ 141 ​ ​ 5,490 2022 ​ 3,552 ​ ​ 1,894 ​ ​ — ​ ​ 5,446 2023 ​ 3,650 ​ ​ 1,894 ​ ​ — ​ ​ 5,544 2024 ​ 4,146 ​ ​ 1,894 ​ ​ — ​ ​ 6,040 Thereafter ​ 20,745 ​ ​ 13,100 ​ ​ — ​ ​ 33,845 Total lease payments $ 37,245 ​ $ 21,623 ​ $ 202 ​ $ 59,070 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Less: amount of lease payments representing interest payments ​ ( 13,790 ) ​ ​ ( 9,498 ) ​ ​ ( 8 ) ​ ​ ( 23,296 ) Present value of lease payments ​ 23,455 ​ ​ 12,125 ​ ​ 194 ​ ​ 35,774 Less: current operating lease liabilities ​ ( 3,407 ) ​ ​ ( 1,894 ) ​ ​ ( 194 ) ​ ​ ( 5,495 ) Non-current operating lease liabilities $ 20,048 ​ $ 10,231 ​ $ — ​ $ 30,279 ​ ( 1) Payments are due in EUR and have been translated at the foreign exchange rate as of June 30, 2020, of $ 1.12 / €1.00). ​ 14 Table of Contents 6 Accrued expenses and other current liabilities ​ Accrued expenses and other current liabilities include the following items: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2020 2019 ​ ​ (in thousands) Accruals for services provided by vendors-not yet billed ​ $ 6,629 ​ $ 5,425 Personnel related accruals and liabilities ​ ​ 6,621 ​ ​ 7,032 Total ​ $ 13,250 ​ $ 12,457 ​ ​ ​ 7 Long-term debt ​ On June 14, 2013, the Company entered into a venture debt loan facility with Hercules Growth Capital, Inc. (“Hercules”), which was amended and restated on June 26, 2014, and again on May 6, 2016 (“2016 Amended Facility”). On December 6, 2018, the Company signed an amendment to the Second Amended and Restated Loan and Security Agreement that both refinanced the then-existing $ 20 million 2016 Amended Facility and provided an additional unconditional commitment of $ 15 million as well as a conditional commitment of $ 15 million that expired on June 30, 2020 (the “2018 Amended Facility”). At signing of the 2018 Amended Facility, the Company drew down an additional $ 15 million for a total of $ 35 million outstanding. The 2018 Amended Facility extended the loan’s maturity date from May 1, 2020 until June 1, 2023. The interest-only period was initially extended from November 2018 to January 1, 2021 and was further extended to January 1, 2022 as a result of meeting the provision in the 2018 Amended Facility of raising more than $ 90.0 million in equity financing in September 2019. The Company is required to repay the facility in equal monthly installments of principal and interest between the end of the interest-only period and the maturity date. The interest rate continues to be adjustable and is the greater of (i) 8.85 % and (ii) 8.85 % plus the prime rate less 5.50 % per annum. Under the 2018 Amended Facility, the Company paid a facility fee of 0.50 % of the $ 35 million outstanding as of signing and owes a back-end fee of 4.95 % of the outstanding debt. In addition, in May 2020 the Company paid a back-end fee of $ 1.0 million in relation to the 2016 Amended Facility. The amortized cost (including interest due presented as part of accrued expenses and other current liabilities) of the 2018 Amended Facility was $ 35.6 million as of June 30, 2020, compared to $ 36.3 million as of December 31, 2019, and is recorded net of discount and debt issuance costs. The foreign currency gain on the loan in the three and six months ended June 30, 2020, was $ 0.7 million and $ 0.0 million compared to a foreign currency gain of $ 0.5 million during the three months ended June 30, 2019, and a foreign currency loss of $ 0.2 million during the six months ended June 30, 2019. Interest expense associated with the 2018 Amended Facility during the three and six months ended June 30, 2020 was $ 0.9 million and $ 1.8 million, respectively, compared to $ 0.9 million and $ 1.9 million during the same periods in 2019. As a covenant in the 2018 Amended Facility, the Company has periodic reporting requirements and is required to keep a minimum cash balance deposited in bank accounts in the United States, equivalent to the lesser of (i) 65 % of the outstanding balance of principal due or (ii) 100% of worldwide cash and cash equivalents. This restriction on cash and cash equivalents only relates to the location of the cash and cash equivalents, and such cash and cash equivalents can be used at the discretion of the Company. In combination with other covenants, the 2018 Amended Facility restricts the Company’s ability to, among other things, incur future indebtedness and obtain additional debt financing, to make investments in securities or in other companies, to transfer assets, to perform certain corporate changes, to make loans to employees, officers and directors, and to make dividend payments and other distributions. The Company secured the facilities by directly or indirectly pledging its total assets of $ 385.3 million with the exception of $ 110.0 million of cash and cash equivalents and other current assets held by uniQure N.V. The 2018 Amended Facility contains provisions that include the occurrence of a material adverse effect, as defined therein, which would entitle Hercules to declare all principal, interest and other amounts owed by the Company immediately due and payable. As of June 30, 2020, the Company was in compliance with all covenants and provisions. ​ 15 Table of Contents 8 Share-based compensation ​ The Company’s share-based compensation plans include the 2014 Amended and Restated Share Option Plan (the “2014 Plan”) and inducement grants under Rule 5653(c)(4) of the Nasdaq Global Select Market with terms similar to the 2014 Plan (together the “2014 Plans”). At the annual general meeting of shareholders in June 2018, the Company’s shareholders approved amendments of the 2014 Plan, increasing the shares authorized for issuance by 3,000,000 to a total of 8,601,471 . ​ a) 2014 Plans ​ Share-based compensation expense recognized by classification included in the Consolidated statements of operations and comprehensive loss in relation to the 2014 Plans for the periods indicated below was as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended June 30, ​ Six months ended June 30, ​ 2020 2019 ​ 2020 ​ 2019 ​ ​ (in thousands) ​ (in thousands) Research and development ​ $ 2,889 ​ $ 2,119 ​ $ 5,271 ​ $ 4,099 Selling, general and administrative ​ ​ 2,825 ​ ​ 2,457 ​ ​ 4,783 ​ ​ 4,755 Total ​ $ 5,714 ​ $ 4,576 ​ $ 10,054 ​ $ 8,854 ​ ​ Share-based compensation expense recognized by award type for the periods indicated below was as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended June 30, ​ Six months ended June 30, ​ 2020 2019 ​ 2020 ​ 2019 ​ ​ (in thousands) ​ (in thousands) Award type ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Share options ​ $ 2,746 ​ $ 2,004 ​ $ 4,954 ​ $ 4,080 Restricted share units (“RSUs”) ​ ​ 2,209 ​ ​ 995 ​ ​ 3,653 ​ ​ 1,925 Performance share units (“PSUs”) ​ ​ 759 ​ ​ 1,577 ​ ​ 1,447 ​ ​ 2,849 Total ​ $ 5,714 ​ $ 4,576 ​ $ 10,054 ​ $ 8,854 ​ As of June 30, 2020, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans were: ​ ​ ​ ​ ​ ​ ​ ​ Unrecognized Weighted average ​ share-based remaining ​ ​ compensation ​ period for ​ ​ expense ​ recognition ​ ​ (in thousands) ​ (in years) Award type ​ ​ ​ ​ ​ Share options ​ $ 27,450 ​ 3.00 Restricted share units ​ ​ 15,705 ​ 2.28 Performance share units ​ ​ 3,519 ​ 1.37 Total ​ $ 46,674 ​ 2.63 ​ The Company satisfies the exercise of share options and vesting of RSUs and PSUs through newly issued ordinary shares. ​ 16 Table of Contents Share options ​ The following table summarizes option activity for the six months ended June 30, 2020: ​ ​ ​ ​ ​ ​ ​ ​ ​ Options ​ ​ Number of ​ Weighted average ​ ordinary shares exercise price ​ ​ ​ ​ ​ ​ Outstanding at December 31, 2019 ​ 2,683,104 ​ $ 21.29 Granted ​ 470,327 ​ $ 53.26 Forfeited ​ ( 69,758 ) ​ $ 36.13 Exercised ​ ( 203,940 ) ​ $ 16.68 Outstanding at June 30, 2020 ​ 2,879,733 ​ $ 26.47 Thereof, fully vested and exercisable at June 30, 2020 ​ 1,530,724 ​ $ 16.06 Thereof, outstanding and expected to vest after June 30, 2020 ​ 1,349,009 ​ $ 38.30 ​ ​ ​ ​ ​ ​ ​ Total weighted average grant date fair value of options issued during the period (in $ millions) ​ ​ ​ $ 14.2 Proceeds from option sales during the period (in $ millions) ​ ​ ​ $ 3.4 ​ Share options are priced on the date of grant and, except for certain grants made to non-executive directors, vest over a period of four years , the first 25 % vests after one year from the grant date and the remainder vests in equal quarterly installments, over years two, three and four. Any options that vest must be exercised by the tenth anniversary of the grant date. The fair value of each option issued is estimated at the respective grant date using the Hull & White option pricing model with the following weighted-average assumptions: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended June 30, Six months ended June 30, Assumptions 2020 2019 2020 2019 Expected volatility ​ 70 % ​ 75 % ​ 70 % ​ 75 % Expected terms ​ 10 years ​ 10 years ​ 10 years ​ 10 years Risk free interest rate ​ 0.76 % - 0.83 % ​ 2.17 % - 2.63 % ​ 0.76 % - 1.44 % ​ 2.17 % - 2.87 % Expected dividend yield ​ 0 % ​ 0 % ​ 0 % ​ 0 % ​ Restricted share units (“RSUs”) ​ The following table summarizes the RSUs activity for the six months ended June 30, 2020: ​ ​ ​ ​ ​ ​ ​ ​ ​ RSU ​ ​ Weighted average ​ ​ Number of ​ grant-date fair ​ ​ ordinary shares ​ value Non-vested at December 31, 2019 ​ 370,830 ​ $ 28.62 Granted ​ 244,449 ​ $ 52.81 Vested ​ ( 171,401 ) ​ $ 19.36 Forfeited ​ ( 14,562 ) ​ $ 45.12 Non-vested at June 30, 2020 ​ 429,316 ​ $ 45.53 ​ ​ ​ ​ ​ ​ Total weighted average grant date fair value of RSUs granted during the period (in $ millions) ​ ​ ​ $ 12.9 ​ RSUs vest over one to three years . RSUs granted to non-executive directors vest one year from the date of grant. ​ 17 Table of Contents Performance share units (“PSUs”) ​ The following table summarizes the PSUs activity for the six months ended June 30, 2020: ​ ​ ​ ​ ​ ​ ​ ​ ​ PSU ​ ​ Weighted average ​ ​ Number of ​ grant-date fair ​ ​ ordinary shares ​ value Non-vested at December 31, 2019 ​ 479,422 ​ $ 21.17 Granted ​ 91,003 ​ $ 57.56 Vested ​ ( 354,105 ) ​ $ 17.44 Non-vested at June 30, 2020 ​ 216,320 ​ $ 42.58 ​ ​ ​ ​ ​ ​ Total weighted average grant date fair value of PSUs granted during the period (in $ millions) ​ ​ ​ $ 5.2 ​ In January 2019, the Company awarded PSUs to its executives and certain other members of senior management. These PSUs were earned as of January 2020 based on an assessment by the Company’s board of directors of the level of achievement of agreed upon performance targets through December 31, 2019. PSUs vest after three years . ​ b) Employee Share Purchase Plan (“ESPP”) ​ In June 2018, the Company’s shareholders adopted and approved an ESPP allowing the Company to issue up to 150,000 ordinary shares. The ESPP is intended to qualify under Section 423 of the Internal Revenue Code of 1986. Under the ESPP, employees are eligible to purchase ordinary shares through payroll deductions, subject to any plan limitations. The purchase price of the ordinary shares on each purchase date is equal to 85 % of the lower of the closing market price on the offering date and the closing market price on the purchase date of each three-month offering period. During the six months ended June 30, 2020, 3,005 ordinary shares were issued under the ESPP compared to 4,870 during the same period in 2019. As of June 30, 2020, a total of 135,202 ordinary shares remains available for issuance under the ESPP plan compared to a total of 142,539 as of June 30, 2019. ​ 9 Income taxes ​ Deferred tax assets and deferred tax liabilities are recognized based on the expected future tax consequences of temporary differences between the financial statement carrying amounts and the income tax basis of assets and liabilities, using current statutory rates. A valuation allowance is recorded against deferred tax assets if it is more likely than not that some or all the deferred tax assets will not be realized. The Company reassessed the need for a full valuation allowance in conjunction with entering into the CSL Behring Agreement. Closing of the CSL Behring Agreement is contingent on the successful completion of reviews under the antitrust laws in the United States, Australia, and the United Kingdom, which has not occurred to date. Closing of the transaction is dependent on the timing, extent and result of the regulatory review process. In its assessment of whether or not it was more likely than not that the Company’s deferred tax assets will be realized, the Company considered all relevant facts and circumstances, including in particular similar regulatory reviews as well as the three-year cumulative losses reported by the Company. The Company concluded that it should continue to record a full valuation allowance as of June 30, 2020. As of December 31, 2019, the Company’s valuation allowance amounted to $ 109.9 million. ​ 18 Table of Contents 10 Basic and diluted earnings per share ​ Diluted earnings per share are calculated by adjusting the weighted average number of ordinary shares outstanding, assuming conversion of all potentially dilutive ordinary shares. As the Company has incurred a loss, all potentially dilutive ordinary shares would have an antidilutive effect, if converted, and thus have been excluded from the computation of loss per share. The shares are presented without giving effect to the application of the treasury method or exercise prices that would be above the share price as of June 30, 2020 and June 30, 2019, respectively. In addition, the BMS warrants were not exercisable as of these dates since this would have required the prior designation of Collaboration Targets by BMS. This would generally result in a lower number of potentially dilutive ordinary shares as some stock option grants as well as the BMS warrants would have been excluded. ​ The potentially dilutive ordinary shares are summarized below: ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ 2020 2019 ​ ​ (ordinary shares) BMS warrants ​ 8,060,500 ​ 8,435,000 Stock options under 2014 Plans ​ 2,879,733 ​ 2,943,756 Non-vested RSUs and earned PSUs ​ 645,636 ​ 842,859 Stock options under previous option plan ​ 14,000 ​ 14,000 Employee share purchase plan ​ 254 ​ 596 Total potential dilutive ordinary shares ​ 11,600,123 ​ 12,236,211 ​ ​ ​ ​ ​ 11 Subsequent events ​ None. ​ ​ ​ ​ ​ 19 Table of Contents